JPWO2020072354A5 - - Google Patents
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- Publication number
- JPWO2020072354A5 JPWO2020072354A5 JP2021518190A JP2021518190A JPWO2020072354A5 JP WO2020072354 A5 JPWO2020072354 A5 JP WO2020072354A5 JP 2021518190 A JP2021518190 A JP 2021518190A JP 2021518190 A JP2021518190 A JP 2021518190A JP WO2020072354 A5 JPWO2020072354 A5 JP WO2020072354A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- seq
- acid sequence
- pharmaceutical composition
- amino acid
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024125570A JP2024161415A (ja) | 2018-10-01 | 2024-08-01 | Ｇｍ１ガングリオシドーシスの治療に有用な組成物 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862739811P | 2018-10-01 | 2018-10-01 | |
| US62/739,811 | 2018-10-01 | ||
| US201962835178P | 2019-04-17 | 2019-04-17 | |
| US62/835,178 | 2019-04-17 | ||
| PCT/US2019/053797 WO2020072354A1 (en) | 2018-10-01 | 2019-09-30 | Compositions useful for treating gm1 gangliosidosis |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024125570A Division JP2024161415A (ja) | 2018-10-01 | 2024-08-01 | Ｇｍ１ガングリオシドーシスの治療に有用な組成物 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022512589A JP2022512589A (ja) | 2022-02-07 |
| JP2022512589A5 JP2022512589A5 (https=) | 2022-10-06 |
| JPWO2020072354A5 true JPWO2020072354A5 (https=) | 2022-10-06 |
| JP7534290B2 JP7534290B2 (ja) | 2024-08-14 |
Family
ID=69165478
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021518190A Active JP7534290B2 (ja) | 2018-10-01 | 2019-09-30 | Ｇｍ１ガングリオシドーシスの治療に有用な組成物 |
| JP2024125570A Pending JP2024161415A (ja) | 2018-10-01 | 2024-08-01 | Ｇｍ１ガングリオシドーシスの治療に有用な組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024125570A Pending JP2024161415A (ja) | 2018-10-01 | 2024-08-01 | Ｇｍ１ガングリオシドーシスの治療に有用な組成物 |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US12594327B2 (https=) |
| EP (1) | EP3860659A1 (https=) |
| JP (2) | JP7534290B2 (https=) |
| KR (1) | KR20210071017A (https=) |
| CN (1) | CN113438954A (https=) |
| AU (1) | AU2019351815A1 (https=) |
| BR (1) | BR112021006060A2 (https=) |
| CA (1) | CA3114175A1 (https=) |
| CL (1) | CL2021000798A1 (https=) |
| CO (1) | CO2021005443A2 (https=) |
| IL (1) | IL281923A (https=) |
| MX (1) | MX2021003869A (https=) |
| PE (1) | PE20211596A1 (https=) |
| SA (1) | SA521421639B1 (https=) |
| SG (1) | SG11202102942VA (https=) |
| WO (1) | WO2020072354A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113438954A (zh) | 2018-10-01 | 2021-09-24 | 宾夕法尼亚州大学信托人 | 可用于治疗gm1神经节苷脂病的组合物 |
| MX2022009462A (es) * | 2020-02-02 | 2022-11-10 | Univ Pennsylvania | Composiciones útiles para tratar la gangliosidosis gm1. |
| AR122013A1 (es) * | 2020-05-05 | 2022-08-03 | Univ Duke | Composiciones de virus adenoasociados compatibles entre especies y métodos para su uso |
| CN116926047A (zh) * | 2022-03-31 | 2023-10-24 | 北京锦篮基因科技有限公司 | 功能性β-半乳糖苷酶变体、AAV介导的人β-半乳糖苷酶表达载体及其用途 |
| WO2024040258A2 (en) * | 2022-08-19 | 2024-02-22 | Codexis, Inc. | Engineered beta- galactosidase polypeptides |
| CN120035662A (zh) * | 2022-10-12 | 2025-05-23 | 新型医药公司 | 用于治疗、预防或改善GM1神经节苷脂沉积症或Morquio综合征B的药物组合物及其施用方法 |
Family Cites Families (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| WO1998046728A1 (en) | 1997-04-14 | 1998-10-22 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| WO1999061643A1 (en) | 1998-05-27 | 1999-12-02 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP1660970A4 (en) | 2003-08-01 | 2007-02-14 | Dna Twopointo Inc | SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| PL3252161T3 (pl) * | 2007-06-06 | 2022-03-07 | Genzyme Corporation | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| AU2013243948A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| CN105377039A (zh) | 2013-05-15 | 2016-03-02 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| BR112017005892A2 (pt) | 2014-09-24 | 2017-12-12 | Hope City | variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma |
| CA3019315A1 (en) | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
| WO2017075338A2 (en) | 2015-10-29 | 2017-05-04 | Voyager Therapeutics, Inc. | Delivery of central nervous system targeting polynucleotides |
| US20190060359A1 (en) | 2015-11-02 | 2019-02-28 | Neuraltus Pharmaceuticals, Inc. | Treatment of neurodegenerative disease with sodium chlorite |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| EP3411488A1 (en) | 2016-02-03 | 2018-12-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| PL3411484T3 (pl) | 2016-02-05 | 2024-02-19 | Emory University | Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego |
| WO2018045347A1 (en) | 2016-09-02 | 2018-03-08 | Spark Therapeutics, Inc. | Methods and vectors for treating cns disorders |
| EP3541946A1 (en) | 2016-11-15 | 2019-09-25 | Regents Of The University Of Minnesota | Method for improving neurological function in mpsi and mpsii and other neurological disorders |
| JP7455579B2 (ja) | 2017-02-28 | 2024-03-26 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
| EP3794131A4 (en) | 2018-05-15 | 2022-01-26 | University of Massachusetts | RAAV VECTORS ENCODING FOR LYSOSOMAL BETA-GALACTOSIDASE (GLB1) AND CATHEPSIN A |
| CN113438954A (zh) | 2018-10-01 | 2021-09-24 | 宾夕法尼亚州大学信托人 | 可用于治疗gm1神经节苷脂病的组合物 |
| US12448629B2 (en) | 2018-10-05 | 2025-10-21 | University Of Massachusetts | rAAV vectors for the treatment of GM1 and GM2 gangliosidosis |
| US12454701B2 (en) | 2018-12-05 | 2025-10-28 | Abeona Therapeutics Inc. | Recombinant adeno-associated viral vector for gene delivery |
| MX2022009462A (es) | 2020-02-02 | 2022-11-10 | Univ Pennsylvania | Composiciones útiles para tratar la gangliosidosis gm1. |
-
2019
- 2019-09-30 CN CN201980079543.8A patent/CN113438954A/zh active Pending
- 2019-09-30 EP EP19836625.4A patent/EP3860659A1/en active Pending
- 2019-09-30 KR KR1020217012705A patent/KR20210071017A/ko active Pending
- 2019-09-30 MX MX2021003869A patent/MX2021003869A/es unknown
- 2019-09-30 SG SG11202102942VA patent/SG11202102942VA/en unknown
- 2019-09-30 AU AU2019351815A patent/AU2019351815A1/en active Pending
- 2019-09-30 BR BR112021006060-0A patent/BR112021006060A2/pt unknown
- 2019-09-30 CA CA3114175A patent/CA3114175A1/en active Pending
- 2019-09-30 US US17/281,378 patent/US12594327B2/en active Active
- 2019-09-30 JP JP2021518190A patent/JP7534290B2/ja active Active
- 2019-09-30 PE PE2021000431A patent/PE20211596A1/es unknown
- 2019-09-30 WO PCT/US2019/053797 patent/WO2020072354A1/en not_active Ceased
-
2021
- 2021-03-29 CL CL2021000798A patent/CL2021000798A1/es unknown
- 2021-03-31 IL IL281923A patent/IL281923A/en unknown
- 2021-04-01 SA SA521421639A patent/SA521421639B1/ar unknown
- 2021-04-27 CO CONC2021/0005443A patent/CO2021005443A2/es unknown
-
2024
- 2024-08-01 JP JP2024125570A patent/JP2024161415A/ja active Pending
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